Nucleic acids adapted for tissue specific expression

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A61K48/0058

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AHUMAN NECESSITIES A61Medical science A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

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A61K48/0058Nucleic acids adapted for tissue specific expression

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Patents Grantslast 30 patents

Information Patent Grant
Drug and gene therapy to treat high myopia and other ocular disorde...
- Patent number 12,233,050
- Issue date Feb 25, 2025
- City University of Hong Kong
- Wenjun Xiong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic constructs for treating cancer
- Patent number 12,233,135
- Issue date Feb 25, 2025
- PRECISION MOLECULAR INC.
- Christopher Ullman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Myosin 15 promoters and uses thereof
- Patent number 12,233,136
- Issue date Feb 25, 2025
- Decibel Therapeutics, Inc.
- Joseph Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
GLP-1 and use thereof in compositions for treating metabolic diseases
- Patent number 12,221,615
- Issue date Feb 11, 2025
- The Trustees of the University of Pennsylvania
- Christian Hinderer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic applications of CPF1-based genome editing
- Patent number 12,214,056
- Issue date Feb 4, 2025
- Duke University
- Charles A. Gersbach
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Optimized human clotting factor IX gene expression cassettes and th...
- Patent number 12,209,263
- Issue date Jan 28, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Messenger RNA therapy for the treatment of ornithine transcarbamyla...
- Patent number 12,201,677
- Issue date Jan 21, 2025
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for retinitis pigmentosa
- Patent number 12,201,698
- Issue date Jan 21, 2025
- Genzyme Corporation
- Catherine O'Riordan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus compositions for restoring HBB gene function...
- Patent number 12,201,657
- Issue date Jan 21, 2025
- City of Hope
- Saswati Chatterjee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and processes for targeted delivery, expression and mo...
- Patent number 12,195,746
- Issue date Jan 14, 2025
- Combined Therapeutics, Inc.
- Romain Micol
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Materials and methods for treatment of hereditary haemochromatosis
- Patent number 12,186,406
- Issue date Jan 7, 2025
- CRISPR THERAPEUTICS AG
- Roman Lvovitch Bogorad
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Formulations for small intestinal delivery of RSV and norovirus ant...
- Patent number 12,186,407
- Issue date Jan 7, 2025
- Vaxart, Inc.
- George Trager
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Systemic gene replacement therapy for treatment of X-linked myotubu...
- Patent number 12,178,856
- Issue date Dec 31, 2024
- Wake Forest University Health Sciences Genethon
- Martin K. Childers
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and compositions relating to the treatment of tumors
- Patent number 12,178,845
- Issue date Dec 31, 2024
- The General Hospital Corporation
- Gary J. Brenner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Hybrid regulatory elements
- Patent number 12,171,843
- Issue date Dec 24, 2024
- Genethon
- Federico Mingozzi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,173,314
- Issue date Dec 24, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Reducing abnormal accumulation of TDP-43 in motor neurons in amyotr...
- Patent number 12,161,728
- Issue date Dec 10, 2024
- Macquarie University
- Stephanie Rayner
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized expression cassettes for gene therapy
- Patent number 12,151,001
- Issue date Nov 26, 2024
- Tenaya Therapeutics, Inc.
- Huanyu Zhou
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Chimeric protein and related genic transfer technology
- Patent number 12,138,319
- Issue date Nov 12, 2024
- Lucio Pastore
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods for treating or alleviating a bone-loss related disease or...
- Patent number 12,133,896
- Issue date Nov 5, 2024
- Kaohsiung Medical University
- Chau-Zen Wang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy delivery of Parkin mutants having increased activity t...
- Patent number 12,133,897
- Issue date Nov 5, 2024
- NysnoBio GT Neurology, LLC
- Jennifer Johnston
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Compositions for treating pathological calcification conditions, an...
- Patent number 12,128,087
- Issue date Oct 29, 2024
- Yale University
- Demetrios Braddock
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Recombinant adeno associated virus encoding clarin-1 and uses thereof
- Patent number 12,129,287
- Issue date Oct 29, 2024
- President and Fellows of Harvard College
- David P. Corey
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Rescuing voltage-gated sodium channel function in inhibitory neurons
- Patent number 12,121,563
- Issue date Oct 22, 2024
- Allen Institute
- John K. Mich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods and materials for GALGT2 gene therapy
- Patent number 12,121,595
- Issue date Oct 22, 2024
- Research Institute at Nationwide Children's Hospital
- Paul Taylor Martin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Non-invasive optogenetic stimulation method to regulate glucose met...
- Patent number 12,121,743
- Issue date Oct 22, 2024
- Albert Einstein College of Medicine
- Young-Hwan Jo
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Capsid variants and methods of using the same
- Patent number 12,116,385
- Issue date Oct 15, 2024
- DYNO THERAPEUTICS, INC.
- Ina Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for recessive dystrophic epidermolysis bullosa using g...
- Patent number 12,110,504
- Issue date Oct 8, 2024
- The Board of Trustees of the Leland Stanford Junior University
- Zurab Siprashvili
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Models of tauopathy
- Patent number 12,110,502
- Issue date Oct 8, 2024
- REGENERON PHARMACEUTICALS, INC.
- Marine Prissette
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus vectors for treatment of Rett syndrome
- Patent number 12,104,163
- Issue date Oct 1, 2024
- Sarepta Therapeutics, Inc.
- Colin O'Banion
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
CAPSID VARIANTS AND METHODS OF USING THE SAME
- Publication number 20250066428
- Publication date Feb 27, 2025
- DYNO THERAPEUTICS, INC.
- Ina Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY METHODS FOR TREATMENT OF DIABETES
- Publication number 20250064978
- Publication date Feb 27, 2025
- Jaguar Gene Therapy, LLC
- Michael Hughes
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Nucleic Acid-Based Compositions and Methods for Treating Small Vess...
- Publication number 20250064977
- Publication date Feb 27, 2025
- The Schepens Eye Research Institute, Inc.
- Joseph F. Arboleda-Velasquez
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
SELF-ASSEMBLING VIRUS-LIKE PARTICLES FOR DELIVERY OF PRIME EDITORS...
- Publication number 20250064979
- Publication date Feb 27, 2025
- THE BROAD INSTITUTE, INC.
- David R. Liu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Materials and Methods for Treatment of Duchenne Muscular Dystrophy
- Publication number 20250064980
- Publication date Feb 27, 2025
- VERTEX PHARMACEUTICALS INCORPORATED
- Ami Meda Kabadi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV VECTORS ENCODING BASE EDITORS AND USES THEREOF
- Publication number 20250064981
- Publication date Feb 27, 2025
- THE BROAD INSTITUTE, INC.
- David R. Liu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT AAV VECTORS FOR TREATING GLUTARIC ACIDURIA TYPE I
- Publication number 20250064982
- Publication date Feb 27, 2025
- SHANGHAI VITALGEN BIOPHARMA CO., LTD.
- Lu GUO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING A CARDIAC DISEASE
- Publication number 20250064983
- Publication date Feb 27, 2025
- The Children's Medical Center Corporation
- Vassilios J. Bezzerides
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
AAV GENE THERAPY FOR THE KIDNEY
- Publication number 20250064984
- Publication date Feb 27, 2025
- The University of Bristol
- Moin Ahson Saleem-Uddin
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
TRANSGENIC EXPRESSION CASSETTE FOR TREATING MUSCULAR DYSTROPHY
- Publication number 20250066437
- Publication date Feb 27, 2025
- SHANGHAI MYGT BIOPHARMACEUTICAL LLC
- Mengdi YAN
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITION FOR PROMOTING MIGRATION OF ADULT STEM CELLS USING INCRE...
- Publication number 20250057983
- Publication date Feb 20, 2025
- SOONCHUNHYANG UNIVERSITY INDUSTRY ACADEMY COOPERATION FOUNDATION
- Nayoung Suh
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CELL-TYPE SPECIFIC MEMBRANE FUSION PROTEINS
- Publication number 20250057984
- Publication date Feb 20, 2025
- THE BROAD INSTITUTE, INC.
- Feng ZHANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Compositions and Methods for Treatment of Ocular Disease Associated...
- Publication number 20250057987
- Publication date Feb 20, 2025
- 4D MOLECULAR THERAPEUTICS INC.
- Christian BURNS
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
REEXPRESSION OF HNF4A TO ALLEVIATE CANCER-ASSOCIATED CACHEXIA
- Publication number 20250057985
- Publication date Feb 20, 2025
- Yeda Research and Development Co. Ltd.
- Ayelet EREZ
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VIRAL VECTORS AND USES THEREOF
- Publication number 20250057986
- Publication date Feb 20, 2025
- THE BROAD INSTITUTE, INC.
- Benjamin E. Deverman
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
IN VIVO REPROGRAMMING OF PHOTORECEPTOR CELLS
- Publication number 20250049956
- Publication date Feb 13, 2025
- CENTRE FOR EYE RESEARCH AUSTRALIA LTD
- Ching Bong WONG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY DELIVERY OF PARKIN MUTANTS HAVING INCREASED ACTIVITY T...
- Publication number 20250049958
- Publication date Feb 13, 2025
- NysnoBio GT Neurology, LLC
- Jennifer JOHNSTON
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
CSF TRANSPORT PATHWAY FOR DELIVERY OF AGENTS TO INNER EAR
- Publication number 20250049957
- Publication date Feb 13, 2025
- University of Rochester
- Maiken Nedergaard
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
- Publication number 20250049959
- Publication date Feb 13, 2025
- Tenaya Therapeutics, Inc.
- Huanyu ZHOU
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-TFR:ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINA...
- Publication number 20250049896
- Publication date Feb 13, 2025
- Regeneron Pharmaceuticals, Inc.
- Maria Praggastis
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Multicomponent systems for site-specific genome modifications
- Publication number 20250049960
- Publication date Feb 13, 2025
- The Regents of the University of California
- Kathleen Collins
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS...
- Publication number 20250049955
- Publication date Feb 13, 2025
- VOYAGER THERAPEUTICS, INC.
- Mathieu Emmanuel NONNENMACHER
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ADENO-ASSOCIATED VIRUS PARTICLES AND METHODS OF USE THEREOF
- Publication number 20250041450
- Publication date Feb 6, 2025
- Insmed Incorporated
- Brian KASPAR
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND COMPOSITIONS FOR TREATING BAG-3 RELATED CARDIOMYOPATHY...
- Publication number 20250041453
- Publication date Feb 6, 2025
- University of Florida Research Foundation, Incorporated
- Barry John Byrne
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ATTENUATED ORTHOMYXOVIRUS-BASED VECTOR PLATFORM
- Publication number 20250043282
- Publication date Feb 6, 2025
- NEW YORK UNIVERSITY
- Benjamin tenOever
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DIS...
- Publication number 20250041455
- Publication date Feb 6, 2025
- Regeneron Pharmaceuticals, Inc.
- Andrew Baik
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RESCUING VOLTAGE-GATED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS
- Publication number 20250041384
- Publication date Feb 6, 2025
- Allen Institute
- John K. Mich
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Gene Therapy of Fibroblast Growth Factor 23 Related Hypophosphatemi...
- Publication number 20250041448
- Publication date Feb 6, 2025
- GENETHON
- Giuseppe RONZITTI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
GENE THERAPY FOR HEARING LOSS
- Publication number 20250041451
- Publication date Feb 6, 2025
- Perceive Biotherapeutics, Inc.
- Derek Welsbie
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT AAV VECTORS FOR TREATING NEURODEGENERATIVE DISORDERS
- Publication number 20250041456
- Publication date Feb 6, 2025
- SHANGHAI VITALGEN BIOPHARMA CO., LTD.
- Yezheng TAO
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Nucleic acids adapted for tissue specific expression

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Drug and gene therapy to treat high myopia and other ocular disorde...

Therapeutic constructs for treating cancer

Myosin 15 promoters and uses thereof

GLP-1 and use thereof in compositions for treating metabolic diseases

Therapeutic applications of CPF1-based genome editing

Optimized human clotting factor IX gene expression cassettes and th...

Messenger RNA therapy for the treatment of ornithine transcarbamyla...

Gene therapy for retinitis pigmentosa

Adeno-associated virus compositions for restoring HBB gene function...

Compositions and processes for targeted delivery, expression and mo...

Materials and methods for treatment of hereditary haemochromatosis

Formulations for small intestinal delivery of RSV and norovirus ant...

Systemic gene replacement therapy for treatment of X-linked myotubu...

Methods and compositions relating to the treatment of tumors

Hybrid regulatory elements

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

Reducing abnormal accumulation of TDP-43 in motor neurons in amyotr...

Optimized expression cassettes for gene therapy

Chimeric protein and related genic transfer technology

Methods for treating or alleviating a bone-loss related disease or...

Gene therapy delivery of Parkin mutants having increased activity t...

Compositions for treating pathological calcification conditions, an...

Recombinant adeno associated virus encoding clarin-1 and uses thereof

Rescuing voltage-gated sodium channel function in inhibitory neurons

Methods and materials for GALGT2 gene therapy

Non-invasive optogenetic stimulation method to regulate glucose met...

Capsid variants and methods of using the same

Gene therapy for recessive dystrophic epidermolysis bullosa using g...

Models of tauopathy

Adeno-associated virus vectors for treatment of Rett syndrome

Patents Applicationslast 30 patents

CAPSID VARIANTS AND METHODS OF USING THE SAME

GENE THERAPY METHODS FOR TREATMENT OF DIABETES

Nucleic Acid-Based Compositions and Methods for Treating Small Vess...

SELF-ASSEMBLING VIRUS-LIKE PARTICLES FOR DELIVERY OF PRIME EDITORS...

Materials and Methods for Treatment of Duchenne Muscular Dystrophy

AAV VECTORS ENCODING BASE EDITORS AND USES THEREOF

RECOMBINANT AAV VECTORS FOR TREATING GLUTARIC ACIDURIA TYPE I

COMPOSITIONS AND METHODS FOR TREATING A CARDIAC DISEASE

AAV GENE THERAPY FOR THE KIDNEY

TRANSGENIC EXPRESSION CASSETTE FOR TREATING MUSCULAR DYSTROPHY

COMPOSITION FOR PROMOTING MIGRATION OF ADULT STEM CELLS USING INCRE...

CELL-TYPE SPECIFIC MEMBRANE FUSION PROTEINS

Compositions and Methods for Treatment of Ocular Disease Associated...

REEXPRESSION OF HNF4A TO ALLEVIATE CANCER-ASSOCIATED CACHEXIA

ADENO-ASSOCIATED VIRAL VECTORS AND USES THEREOF

IN VIVO REPROGRAMMING OF PHOTORECEPTOR CELLS

GENE THERAPY DELIVERY OF PARKIN MUTANTS HAVING INCREASED ACTIVITY T...

CSF TRANSPORT PATHWAY FOR DELIVERY OF AGENTS TO INNER EAR

OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY

ANTI-TFR:ACID SPHINGOMYELINASE FOR TREATMENT OF ACID SPHINGOMYELINA...

Multicomponent systems for site-specific genome modifications

COMPOSITONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS...

ADENO-ASSOCIATED VIRUS PARTICLES AND METHODS OF USE THEREOF

METHODS AND COMPOSITIONS FOR TREATING BAG-3 RELATED CARDIOMYOPATHY...

ATTENUATED ORTHOMYXOVIRUS-BASED VECTOR PLATFORM

ANTI-TFR:GAA AND ANTI-CD63:GAA INSERTION FOR TREATMENT OF POMPE DIS...

RESCUING VOLTAGE-GATED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS

Gene Therapy of Fibroblast Growth Factor 23 Related Hypophosphatemi...

GENE THERAPY FOR HEARING LOSS

RECOMBINANT AAV VECTORS FOR TREATING NEURODEGENERATIVE DISORDERS